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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/ct2/show/NCT02093897
Registration number
NCT02093897
Ethics application status
Date submitted
19/03/2014
Date registered
21/03/2014
Date last updated
27/01/2017
Titles & IDs
Public title
Pharmacokinetic, Efficacy, and Safety Study of Recombinant Factor VIII Single Chain (rVIII-SingleChain) in Children With Severe Hemophilia A
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Scientific title
A Phase III Open-label Pharmacokinetic, Efficacy and Safety Study of rVIII-SingleChain in a Pediatric Population With Severe Hemophilia A
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Secondary ID [1]
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2012-001336-65
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Secondary ID [2]
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CSL627_3002
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Universal Trial Number (UTN)
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Trial acronym
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Congenital Hemophilia A
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Condition category
Condition code
Blood
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Clotting disorders
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Human Genetics and Inherited Disorders
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Other human genetics and inherited disorders
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Intervention/exposure
Study type
Interventional
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Description of intervention(s) / exposure
Other interventions - rVIII-SingleChain
Experimental: rVIII-SingleChain - Subjects will be assigned to either an on-demand or prophylaxis regimen and will receive rVIII-SingleChain as an intravenous (IV) infusion. Subjects assigned to a prophylaxis regimen will be treated with 15 to 50 IU/kg of rVIII-SingleChain every second day or 2 to 3 times per week, or at the investigator's discretion, based on available PK data, the FVIII treatment regimen used before enrollment and/or the subject's bleeding phenotype. The dose for on-demand treatment of a bleeding episode is based on the recommendations of the World Federation of Hemophilia (WFH), with a minimum dose of 15 IU/kg. All subjects were to be treated for a minimum of 50 EDs. For the PK evaluation, the subjects will receive a single IV dose of 50 IU/kg of rVIII-SingleChain on Day 1 at the start of the PK evaluation period.
Other interventions: rVIII-SingleChain
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Intervention code [1]
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Other interventions
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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Treatment Success
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Assessment method [1]
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Rate of treatment success where treatment success of a bleeding episode is defined as a rating of "excellent" or "good" based on the investigator's overall clinical assessment of hemostatic efficacy (using a 4-point scale of excellent, good, moderate or poor/no response) on the on-demand and prophylaxis regimens combined. The rate of success was based on the number of treated bleeding events; there were 347 treated bleeding events in the Efficacy Population.
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Timepoint [1]
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Up to 1 year
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Secondary outcome [1]
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Annualized Bleeding Rate
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Assessment method [1]
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The annualized bleeding rate was defined as the number of bleeding episodes requiring treatment divided by the efficacy evaluation period in days, x 365.25, and is presented separately for the on-demand regimen and the prophylaxis regimens.
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Timepoint [1]
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Up to 1 year
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Secondary outcome [2]
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Percentage of Bleeding Episodes Requiring 1, 2, 3, or More Than 3 Infusions of rVIII-SingleChain to Achieve Hemostasis.
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Assessment method [2]
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Timepoint [2]
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Up to 1 year
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Secondary outcome [3]
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Consumption of rVIII-SingleChain - IU/kg Per Subject Per Month
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Assessment method [3]
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Timepoint [3]
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Up to 1 year
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Secondary outcome [4]
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Consumption of rVIII-SingleChain - IU/kg Per Subject Per Year
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Assessment method [4]
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Timepoint [4]
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Up to 1 year
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Secondary outcome [5]
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Consumption of rVIII-SingleChain - IU/kg Per Bleeding Event
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Assessment method [5]
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Timepoint [5]
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Up to 1 year
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Secondary outcome [6]
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Consumption of rVIII-SingleChain (On-demand Regimen) - Number of Infusions Per Subject Per Month
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Assessment method [6]
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Timepoint [6]
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Up to 1 year
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Secondary outcome [7]
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Consumption of rVIII-SingleChain (On-demand Regimen) - Number of Infusions Per Subject Per Year
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Assessment method [7]
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Timepoint [7]
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Up to 1 year
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Secondary outcome [8]
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Incremental Recovery
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Assessment method [8]
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Incremental recovery expressed as (IU/dL)/(IU/kg) corrected for subject's predose plasma FVIII activity measured using the chromogenic substrate assay.
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Timepoint [8]
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At 1 hour after the start of infusion
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Secondary outcome [9]
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Half-life (t1/2) of rVIII-SingleChain
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Assessment method [9]
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Half-life (t1/2) of rVIII-SingleChain, baseline uncorrected; plasma FVIII activity measured using the chromogenic substrate assay.
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Timepoint [9]
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Immediately before dosing, and at approximately 1, 5, 10, 24, and 48 hours after dosing.
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Secondary outcome [10]
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Area Under the Concentration Curve (AUC)
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Assessment method [10]
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AUC to the last sample with quantifiable drug concentration (AUC0-t), baseline uncorrected; plasma FVIII activity measured using the chromogenic substrate assay.
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Timepoint [10]
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Immediately before dosing, and at approximately 1, 5, 10, 24, and 48 hours after dosing.
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Secondary outcome [11]
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Clearance (Cl) of rVIII-SingleChain
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Assessment method [11]
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Clearance (Cl) of rVIII-SingleChain, baseline uncorrected; plasma FVIII activity measured using the chromogenic substrate assay.
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Timepoint [11]
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Immediately before dosing, and at approximately 1, 5, 10, 24, and 48 hours after dosing.
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Secondary outcome [12]
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Number of Subjects With Inhibitor Formation to rVIII-SingleChain
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Assessment method [12]
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The number of subjects who develop inhibitors to rVIII-SingleChain, defined as a rVIII-SingleChain antibody titer of at least 0.6 Bethesda Units (BU) per mL after receiving study drug.
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Timepoint [12]
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At screening, then after dosing at approximately monthly intervals for 6 months, then every 3 months until reaching 50 EDs, and at the end of study visit (up to approximately 12 months).
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Eligibility
Key inclusion criteria
- Diagnosis of severe hemophilia A defined as < 1% Factor VIII (FVIII) concentration
(FVIII:C) documented in medical records,
- Males < 12 years of age,
- Subjects who have received > 50 EDs with a FVIII product,
- Prior PK data (at least incremental recovery and half-life) from previous FVIII
exposure for subjects participating in the PK part
- Investigator believes that the subject is willing and able to adhere to all protocol
requirements. Investigator believes that the subject's parent(s) or legally acceptable
representative(s) is / are willing and able to adhere to all protocol requirements.
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Minimum age
No limit
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Maximum age
11
Years
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Sex
Males
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Can healthy volunteers participate?
No
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Key exclusion criteria
- Any history of or current FVIII inhibitors
- Use of an Investigational Medical Product (IMP) within 30 days prior to the first
rVIII-SingleChain administration,
- Administration of any cryoprecipitate, whole blood or plasma within 30 days prior to
administration of rVIII-SingleChain,
- Known hypersensitivity (allergic reaction or anaphylaxis) to any FVIII product or
hamster protein,
- Subject currently receiving IV immunomodulating agents such as immunoglobulin or
chronic systemic corticosteroid treatment,
- Subject with serum aspartate aminotransferase (AST) or serum alanine aminotransferase
(ALT) values >5 times (x) the upper limit of normal (ULN) at Screening,
- Subjects with serum creatinine values >2 x ULN at Screening,
- Evidence of thrombosis, including deep vein thrombosis, stroke, pulmonary embolism,
myocardial infarction and arterial embolus within 3 months before Day 1,
- Experienced life-threatening bleeding episode or had major surgery or an orthopedic
surgical procedure during the 3 months before rVIII-SingleChain administration.
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Study design
Purpose of the study
Treatment
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Allocation to intervention
N/A
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Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
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Methods used to generate the sequence in which subjects will be randomised (sequence generation)
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Masking / blinding
Open (masking not used)
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Who is / are masked / blinded?
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Intervention assignment
Single group
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Other design features
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Phase
Phase 3
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Type of endpoint/s
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Statistical methods / analysis
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Recruitment
Recruitment status
Completed
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Data analysis
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Reason for early stopping/withdrawal
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Other reasons
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Date of first participant enrolment
Anticipated
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Actual
1/03/2014
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Date of last participant enrolment
Anticipated
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Actual
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Date of last data collection
Anticipated
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Actual
1/08/2015
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Sample size
Target
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Accrual to date
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Final
84
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Recruitment in Australia
Recruitment state(s)
VIC
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Recruitment hospital [1]
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Study Site - Melbourne
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Recruitment postcode(s) [1]
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3052 - Melbourne
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Recruitment outside Australia
Country [1]
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United States of America
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State/province [1]
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Colorado
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United States of America
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State/province [2]
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Illinois
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Austria
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State/province [3]
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Linz
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Austria
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State/province [4]
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Vienna
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France
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State/province [5]
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Brest
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France
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State/province [6]
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Le Kremlin Bicetre
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France
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Lille Cedex
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France
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Nantes
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France
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Paris
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Georgia
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Tbilisi
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Germany
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Bonn
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Germany
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State/province [12]
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Bremen
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Germany
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Frankfurt/Main
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Germany
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Hannover
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Italy
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Milano
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Lebanon
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Beirut
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Malaysia
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Kuala Lumpur
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Netherlands
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Amsterdam
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Netherlands
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Njmegen
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Netherlands
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Utrecht
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Philippines
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Cebu City
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Philippines
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Davao City
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Poland
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Rzeszow
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Portugal
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Oporto
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Romania
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Timisoara
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Spain
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Madrid
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Switzerland
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Lucerne
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Thailand
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Bangkok
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Thailand
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Chiang Mai
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Thailand
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Khon Kaen
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Thailand
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Songkla
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Turkey
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Adana
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Turkey
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Istanbul
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Turkey
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Izmir
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Ukraine
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Lviv
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Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
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Name
CSL Behring
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Address
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Ethics approval
Ethics application status
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Summary
Brief summary
This is an international, multicenter, open-label study to assess the efficacy, safety, and
pharmacokinetic (PK) profile of rVIII-SingleChain in pediatric patients with severe
hemophilia A. A minimum of 25 previously treated subjects = 6 to < 12 years of age and at
least 25 subjects < 6 years of age who have undergone > 50 exposure days (EDs) with a
previous Factor VIII (FVIII) product are planned to be enrolled. Subjects will be assigned to
either an on-demand or prophylaxis treatment regimen and will receive rVIII-SingleChain at a
dose to be determined by the investigator. Hemostatic efficacy will be assessed by the
subject/caregiver and the investigator who will assess overall efficacy by a 4-point scale.
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Trial website
https://clinicaltrials.gov/ct2/show/NCT02093897
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Trial related presentations / publications
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Public notes
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Contacts
Principal investigator
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Program Director
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Address
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CSL Behring
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Phone
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Fax
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Email
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Contact person for public queries
Name
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Address
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Fax
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Contact person for scientific queries
Summary Results
For IPD and results data, please see
https://clinicaltrials.gov/ct2/show/NCT02093897
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